RU2019140862A - Варианты сульфамидазы (sgsh), векторы, композиции и способы, и применения для лечения мукополисахаридоз типа iiia (mps iiia) - Google Patents
Варианты сульфамидазы (sgsh), векторы, композиции и способы, и применения для лечения мукополисахаридоз типа iiia (mps iiia) Download PDFInfo
- Publication number
- RU2019140862A RU2019140862A RU2019140862A RU2019140862A RU2019140862A RU 2019140862 A RU2019140862 A RU 2019140862A RU 2019140862 A RU2019140862 A RU 2019140862A RU 2019140862 A RU2019140862 A RU 2019140862A RU 2019140862 A RU2019140862 A RU 2019140862A
- Authority
- RU
- Russia
- Prior art keywords
- composition
- component
- composition according
- cystitis
- kda
- Prior art date
Links
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0083—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the administration regime
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y310/00—Hydrolases acting on sulfur-nitrogen bonds (3.10)
- C12Y310/01—Hydrolases acting on sulfur-nitrogen bonds (3.10) acting on sulfur-nitrogen bonds (3.10.1)
- C12Y310/01001—N-Sulfoglucosamine sulfohydrolase (3.10.1.1)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- General Chemical & Material Sciences (AREA)
- Neurosurgery (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Immunology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762505423P | 2017-05-12 | 2017-05-12 | |
| US62/505,423 | 2017-05-12 | ||
| PCT/US2018/032454 WO2018209317A1 (en) | 2017-05-12 | 2018-05-11 | Sulfamidase (sgsh) variants, vectors, compositions and methods and uses for treating mucopolysaccharidosis type iiia (mps iiia) |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| RU2019140862A true RU2019140862A (ru) | 2021-06-15 |
| RU2019140862A3 RU2019140862A3 (enExample) | 2021-08-17 |
Family
ID=64104969
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2019140862A RU2019140862A (ru) | 2017-05-12 | 2018-05-11 | Варианты сульфамидазы (sgsh), векторы, композиции и способы, и применения для лечения мукополисахаридоз типа iiia (mps iiia) |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US12221635B2 (enExample) |
| EP (1) | EP3622060A4 (enExample) |
| JP (1) | JP7196099B2 (enExample) |
| CN (1) | CN110809626A (enExample) |
| AU (1) | AU2018265869C1 (enExample) |
| BR (1) | BR112019023869A2 (enExample) |
| CA (1) | CA3061925A1 (enExample) |
| MX (1) | MX2019013528A (enExample) |
| RU (1) | RU2019140862A (enExample) |
| WO (1) | WO2018209317A1 (enExample) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN108728495A (zh) * | 2018-05-31 | 2018-11-02 | 深圳市免疫基因治疗研究院 | 一种Sanfilippo A综合症慢病毒载体、慢病毒及其制备方法和应用 |
| ES3035673T3 (en) * | 2018-12-20 | 2025-09-08 | Esteve Pharmaceuticals Sa | Recombinant vectors for the long term treatment of mucchopolysacharidosis |
| WO2021011841A1 (en) * | 2019-07-18 | 2021-01-21 | Lysogene | Compositions and methods for the treatment of sanfilippo disease and other disorders |
| CN115023242B (zh) | 2019-11-22 | 2025-03-14 | 费城儿童医院 | 腺相关病毒载体变体 |
| KR20220153021A (ko) * | 2020-03-11 | 2022-11-17 | 리모터 테라퓨틱스, 인코포레이티드 | 중추신경계를 통한 단백질을 확산시키기 위한 방법 및 재료 |
| CN111718947B (zh) * | 2020-06-18 | 2022-08-23 | 舒泰神（北京）生物制药股份有限公司 | 用于治疗ⅲa或ⅲb型粘多糖贮积症的腺相关病毒载体及用途 |
| AU2022388800A1 (en) * | 2021-11-12 | 2024-06-27 | The Trustees Of The University Of Pennsylvania | Gene therapy for treatment of mucopolysaccharidosis iiia |
| US20250034533A1 (en) * | 2021-11-12 | 2025-01-30 | Amicus Therapeutics, Inc. | Compositions And Methods For Treating Mucopolysaccharidosis IIIA |
| WO2023204202A1 (ja) * | 2022-04-19 | 2023-10-26 | Ｊｃｒファーマ株式会社 | マンノース－６－リン酸の数を減少させた糖蛋白質 |
| CN115029360B (zh) * | 2022-05-30 | 2024-08-02 | 上海勉亦生物科技有限公司 | 用于治疗粘多糖贮积症iiia型的转基因表达盒 |
| CN120379648A (zh) * | 2022-12-23 | 2025-07-25 | 株式会社绿十字 | 具有改善的稳定性的包含乙酰肝素n-硫酸酯酶的药物组合物 |
| WO2026162810A1 (en) * | 2025-02-03 | 2026-08-06 | Institut National de la Santé et de la Recherche Médicale | Methods for the intravenous treatment of sanfilippo syndrome type iiia |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5863782A (en) * | 1995-04-19 | 1999-01-26 | Women's And Children's Hospital | Synthetic mammalian sulphamidase and genetic sequences encoding same |
| EP2550359B1 (en) * | 2010-03-23 | 2017-08-09 | Intrexon Corporation | Vectors conditionally expressing therapeutic proteins, host cells comprising the vectors, and uses thereof |
| EP2394667A1 (en) | 2010-06-10 | 2011-12-14 | Laboratorios Del Dr. Esteve, S.A. | Vectors and sequences for the treatment of diseases |
| ES2745470T3 (es) * | 2011-03-31 | 2020-03-02 | Univ Iowa Res Found | Partícula de AAV2 que comprende una proteína de la cápside de AAV2 y un vector que comprende un ácido nucleico que codifica una tripeptidilo peptidasa 1 (TPP1) para el tratamiento de lipofuscinosis ceroide infantil tardía (LINCL) en un mamífero no roedor mediante inyección intraventricular o administración icv |
| EP3166626B1 (en) * | 2014-07-11 | 2021-04-28 | Biostrategies LC | Materials and methods for treating disorders associated with sulfatase enzymes |
-
2018
- 2018-05-11 MX MX2019013528A patent/MX2019013528A/es unknown
- 2018-05-11 US US16/611,458 patent/US12221635B2/en active Active
- 2018-05-11 AU AU2018265869A patent/AU2018265869C1/en active Active
- 2018-05-11 EP EP18798587.4A patent/EP3622060A4/en active Pending
- 2018-05-11 JP JP2019562293A patent/JP7196099B2/ja active Active
- 2018-05-11 WO PCT/US2018/032454 patent/WO2018209317A1/en not_active Ceased
- 2018-05-11 CN CN201880043043.4A patent/CN110809626A/zh active Pending
- 2018-05-11 CA CA3061925A patent/CA3061925A1/en active Pending
- 2018-05-11 BR BR112019023869-7A patent/BR112019023869A2/pt active Search and Examination
- 2018-05-11 RU RU2019140862A patent/RU2019140862A/ru unknown
Also Published As
| Publication number | Publication date |
|---|---|
| WO2018209317A1 (en) | 2018-11-15 |
| AU2018265869C1 (en) | 2025-01-30 |
| CA3061925A1 (en) | 2018-11-15 |
| AU2018265869A1 (en) | 2019-11-21 |
| US20210324354A1 (en) | 2021-10-21 |
| RU2019140862A3 (enExample) | 2021-08-17 |
| JP2020519629A (ja) | 2020-07-02 |
| EP3622060A1 (en) | 2020-03-18 |
| EP3622060A4 (en) | 2021-02-17 |
| BR112019023869A2 (pt) | 2020-06-09 |
| JP7196099B2 (ja) | 2022-12-26 |
| MX2019013528A (es) | 2020-07-14 |
| CN110809626A (zh) | 2020-02-18 |
| AU2018265869B2 (en) | 2024-10-24 |
| US12221635B2 (en) | 2025-02-11 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| CA2782122A1 (en) | Compositions and methods for stabilized polysaccharide formulations | |
| WO2009039993A3 (en) | Use of melanocyte-stimulating hormone release-inhibiting factor as a therapeutic agent in the treatment of pseudomonas aeruginosa infection | |
| WO2009043466A3 (en) | Therapeutic use of the peptide gluten exorphin a5 | |
| WO2009043463A3 (en) | Use of gluten exorphin c : as a therapeutic agent | |
| WO2009040068A3 (en) | Use of a peptide as a therapeutic agent | |
| RU2013103534A (ru) | Лечение легочных инфекций с помощью введения тобрамицина путем аэролизации | |
| AU2016228976B2 (en) | Compositions and methods for treating bacterial infection | |
| WO2009046822A3 (en) | Use of the peptide ymdgtmsqv as a therapeutic agent | |
| US20140134140A1 (en) | Mesenchymal stem cell compositions for the treatment of microbial infections | |
| WO2009033712A3 (en) | Use of melanotan ii as a therapeutic agent | |
| WO2009040005A9 (en) | Use of the peptide rfmwmr as a therapeutic agent | |
| RU2011101713A (ru) | Фармацевтическая композиция | |
| WO2009033791A3 (en) | Use of a peptide as a therapeutic agent | |
| WO2009046858A3 (en) | Therapeutic uses of kisspeptin 13 and compositions thereof | |
| FI2034956T4 (fi) | Matalan viskositeetin omaavia glykosaminoglykaaneja käsittäviä koostumuksia ja mainitun koostumuksen käyttö kroonisen kystiitin terapiassa | |
| US20220241301A1 (en) | Compositions and methods for inhibiting inflammation and diseases using an alginic acid-based antimicrobial compound | |
| KR20160008496A (ko) | 헤파리노이드, 급성 작용성 마취제 및 버퍼를 포함하는 안정한 조성물 | |
| FI3972603T3 (fi) | Menetelmiä sjögrenin oireyhtymän hoitamiseksi käyttäen brutonin tyrosiinikinaasin estäjää | |
| JP6474193B2 (ja) | 局所麻酔剤、ヘパリノイド、及び緩衝剤を含む組成物を製造する方法 | |
| JP2009538287A5 (ja) | 低粘度グリコサミノグリカンで構成される組成物及び該組成物の間質性膀胱炎の治療での使用 | |
| DK2734203T3 (en) | A pharmaceutical composition for the treatment of a respiratory disease | |
| BR112014003529A2 (pt) | combinações de corroles e estatinas | |
| JP2022550658A5 (enExample) | ||
| RU2011125599A (ru) | Энергетическая питательная композиция, содержащая слабительное средство, электролиты и углеводы | |
| RU2018125252A (ru) | Синергическая комбинация пирролидонкарбоновой кислоты и/или ее солей и гиалуроновой кислоты и/или ее солей для применения в лечении и/или профилактике сухости и раздражения слизистых оболочек, и связанные фармацевтические композиции |